New NIH Funding and Clinical Trials Start Soon in Effort to Cure SCD

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Developing tools to assess if gene therapy treatments can reduce symptoms or cure sickle cell disease

Dr. Umut Gurkan

Congratulations to Umut Gurkan, PhD for receiving $3.7M in funding from the National Heart, Lung, and Blood Institute (NHLBI) to assess emerging genome-editing based therapies being tested for curing sickle cell disease (SCD). Dr. Gurkan and his team have developed micro-engineered tools to assess the safety and effectiveness of gene therapy treatments in patients with SCD from blood samples before and after the treatment. 

This project could lead to one simple blood test to tell whether a patient is benefitting from any given treatment. A test this simple could be translated to patients across the globe. “The reason the NIH is so focused on curing SCD is that it is the ‘poster child’ for gene-editing efforts,” Dr. Gurkan said. “If we can prove that we can cure an inherited mutation like SCD effectively and safely, then you convince the funders and the public that it is worth the expense and the effort to go after more complex inherited diseases which are less understood.”

Clinical trials will be conducted over the next two years at clinics located at leading research universities, including Stanford University, University of California-San Francisco, Emory University, University of North Carolina, and Albert Einstein College of Medicine. Clinical evaluation of the blood samples will be conducted at Case Western Reserve University, Case School of Engineering.

Original story posted in the Daily.